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Human iPSC Modeling Reveals Mutation-Specific Responses to Gene Therapy in a Genotypically Diverse Dominant Maculopathy.
Sinha, Divya; Steyer, Benjamin; Shahi, Pawan K; Mueller, Katherine P; Valiauga, Rasa; Edwards, Kimberly L; Bacig, Cole; Steltzer, Stephanie S; Srinivasan, Sandhya; Abdeen, Amr; Cory, Evan; Periyasamy, Viswesh; Siahpirani, Alireza Fotuhi; Stone, Edwin M; Tucker, Budd A; Roy, Sushmita; Pattnaik, Bikash R; Saha, Krishanu; Gamm, David M.
Afiliação
  • Sinha D; McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI 53705, USA; Waisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.
  • Steyer B; McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI 53705, USA; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA.
  • Shahi PK; McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI 53705, USA; Department of Pediatrics, University of Wisconsin-Madison, Madison, WI 53792, USA.
  • Mueller KP; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA.
  • Valiauga R; Waisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.
  • Edwards KL; Waisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.
  • Bacig C; Waisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA.
  • Steltzer SS; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA.
  • Srinivasan S; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA.
  • Abdeen A; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA.
  • Cory E; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA.
  • Periyasamy V; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA.
  • Siahpirani AF; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA.
  • Stone EM; Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
  • Tucker BA; Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
  • Roy S; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA; Department of Biostatistics, University of Wisconsin-Madison, Madison, WI 53792, USA.
  • Pattnaik BR; McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI 53705, USA; Department of Pediatrics, University of Wisconsin-Madison, Madison, WI 53792, USA; Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison, Madison, WI 53705, USA.
  • Saha K; McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI 53705, USA; Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI 53715, USA; Department of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI 53706, USA. Electronic address: ks
  • Gamm DM; McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI 53705, USA; Waisman Center, University of Wisconsin-Madison, Madison, WI 53705, USA; Department of Ophthalmology and Visual Sciences, University of Wisconsin-Madison, Madison, WI 53705, USA. Electronic address: dgamm@wisc
Am J Hum Genet ; 107(2): 278-292, 2020 08 06.
Article em En | MEDLINE | ID: mdl-32707085

Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Células-Tronco Pluripotentes Induzidas / Degeneração Macular / Mutação Tipo de estudo: Prognostic_studies Limite: Humans Idioma: En Revista: Am J Hum Genet Ano de publicação: 2020 Tipo de documento: Article País de afiliação: Estados Unidos

Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Células-Tronco Pluripotentes Induzidas / Degeneração Macular / Mutação Tipo de estudo: Prognostic_studies Limite: Humans Idioma: En Revista: Am J Hum Genet Ano de publicação: 2020 Tipo de documento: Article País de afiliação: Estados Unidos